View clinical trials related to Siderosis.
Filter by:Cerebellar superficial siderosis (SS) has been recently reported to be present in about 10% of both hereditary (n=50) and sporadic (n=46) cerebral amyloid angiopathy (CAA) patients on 3T MRI using susceptibility-weighted imaging (SWI) in the majority of patients. In that study, cerebellar SS was associated with a higher number of supratentorial lobar and superficial cerebellar macrobleeds (although cerebellar SS was not directly located adjacent to these cerebellar macrobleeds). It is unclear if cerebellar SS is caused by in situ leakage of cerebellar leptomeningeal vessels or rather represents hemorrhagic diffusion from cerebellar parenchymal micro/macrobleeds or from supratentorial bleeding sources via the tentorium cerebelli (TC).
One in six people in the United Kingdom and over 400 million people worldwide have disabling hearing loss. This figure will double by 2050 as predicted by the World Health Organisation. There is an urgent need to improve our knowledge regarding hearing loss, its underlying mechanisms, optimal diagnostic modalities, reliable and accurate functional and imaging biomarkers. A less-well studied condition associated with progressive hearing loss is infratentorial superficial siderosis (iSS). It results from iron deposition along the surfaces of brain structures which control hearing and balance. It is currently considered uncommon, but may well be under-recognised and therefore under-reported. Despite its severity, our current understanding of its impact on the hearing (auditory) and balance (vestibular) functions is limited, and this has an adverse impact on the treatment offered to these patients. Additionally, iSS patients have been reported to have cognitive impairment yet literature reports of cognitive assessment in iSS are few. The cognitive dysfunction may be specific to iSS or due to progressive hearing impairment or a combination of both, and further studies are required to establish this. Olfaction is also known to be affected in patients with iSS yet is rarely reported in the literature. Due to the significant morbidity and progressive nature, there is a clear need to improve our understanding of the audiovestibular dysfunction resulting from iSS. The aim of this study is to comprehensively assess audiovestibular function in iSS compared to age-related hearing loss and the controls/normative data and as a means to quantify deficits for monitoring disease progression and response to treatment, to assess the impact on the quality of life, to analyse clinically-obtained data (including imaging, cognitive and laboratory data), and correlate these with functional findings in iSS.
Heart failure from myocardial iron deposition is a severe complication for patients with hematological disorders who need repeated blood transfusions. Increased cardiac iron content impacts the contractility of cardiomyocytes and can also lead to myocarditis, pericarditis, and arrhythmias. The severity of cardiac dysfunction depends on the amount of iron deposited in the myocardium. Cardiovascular magnetic resonance (CMR) imaging is used as noninvasive method to evaluate the amount of iron in the heart. Myocardial T2* value has been shown to correlate well with biopsy-derived iron concentration in the heart, and myocardial T2* values less than 20ms (indicating elevated iron) were found to be associated with LV dysfunction and improve in concert with LV function during recovery. The majority of the recent studies about myocardial iron overload and the effect of iron chelation therapy were focused on patients with transfusion-dependent hematological disorder, especially beta-thalassemia major. The objective of this 3-year project is to evaluate myocardial iron deposition in patients with heart failure, induced by variable causes. With myocardial T2* imaging, the investigators will analyze the decreased signal intensity in the ventricular septum and quantitatively acquire the T2* value as marker for myocardial iron deposition. The first year is a cross-sectional study. The investigators aim to compare the severity of myocardial iron deposition of normal subjects and that of stable HF patients in recovery with normal or impaired ejection fraction (EF). Total 60 subjects will be enrolled, with 20 subjects in each group. In the 2nd and 3rd years, the investigators plan a prospective longitudinal study of 40 subjects. Enrolled patients will be evaluated with cardiac T2* imaging at three time points, i.e., disease onset, 6 months and one year after treatment, and will be followed up until the end of this project (1.5~3-year follow up). In total 120 MR scans will be performed in the 2nd and 3rd years. The presence and severity of myocardial iron deposition will be correlated with the disease course, patient biochemistry data and clinical outcome.
Superficial siderosis is a progressive neurological disease caused by iron deposition in the central nervous system (CNS) from chronic subarachnoid bleeding. Until 2011, there has been no effective treatment for this progressive condition that leads to hearing loss, spasticity, weakness, loss of bowel/bladder function, incoordination, dementia and ultimately death. Last year, the investigators demonstrated that a lipid soluble iron chelator, deferiprone, can reduce hemosiderin deposition in patients with superficial siderosis by MRI in as little as 3 months. As the only therapy that can improve this condition, chelation with deferiprone is the standard of care for treatment of superficial siderosis. Now that the FDA has approved deferiprone in the United States for thalassemia, the investigators propose documenting the clinical effect of deferiprone over 2 years in superficial siderosis patients. The investigators' goal is to understand how the clinical course of this disease is altered using standard of care chelation therapy with deferiprone. Patients with superficial siderosis who are taking deferiprone for chelation therapy at doses consistent with the standard of care will be offered enrollment into this observational study. Patients will be treated and monitored locally by participating neurologists who have agreed to help the investigators collect information for this study. The investigators are interested in documenting the clinical effect of deferiprone on hearing, ataxia and myelopathy using standardized scales performed and documenting the effect of deferiprone on hemosiderin deposition in the CNS by MRI, all performed according to standard of care.
This is a 2 visit research study for patients with or without a diagnosis of migraine. Participants will be administered informed consent, have a medical history taken and a physical examination performed, and complete 3 questionnaires at the first visit. The participants will have a functional MRI performed after completion of Visit 1. Study stipends will be given for each completed research visit. The purpose of this study is to potentially identify risk factors and/or biomarkers (which are differences in the brain structures)by comparing the brain MRI scans of migraine sufferers to brain scans of control persons to attempt to identify migraine progression using the functional MRI scans.
This protocol intends to detail investigations necessary to evaluate the ocular safety of subjects enrolled in study A4321001 and who have received formulated Drug Product (Lot 8716-098).